Giving Thanks by Looking Forward

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The CIRM Team gather to give Thanks

Thanksgiving is traditionally a time of reflection, a time to look back and express gratitude for all the good things that have happened in the past year. At CIRM we have a lot to be thankful for but this Thanksgiving we are looking forward, not backward. We’re unveiling our new Strategic Plan, our blueprint for the future, and we would love to hear what you think about it.

Randy Mills, our President and CEO, calls the Strategic Plan “a bold, new vision” for what we hope to achieve over the next five years. After reading it we hope you’ll agree.

Taking it on the road

We actually began this process several months ago with a talking tour of California. Randy Mills went around the state talking to researchers, academics, company officials, patients and patient advocates – anyone who has a stake in what we do. He posed a few simple questions such as: “what’s impeding progress?” and “how do you think we could do better?” and asked them what they thought we should focus on in the next five years.

As you can imagine we got a wide range of answers, but there was also surprising agreement on some key issues – such as the need to push for regulatory reform to help remove some of the obstacles slowing down the ability of researchers to move their therapies into clinical trials.

Bold ideas

The plan is an ambitious one, but then as Sherry Lansing, the cancer Patient Advocate member of our Board, says in a news release, why aim low:

“As we enter what could be CIRM’s last phase, we want to show the people of California that we are doing everything we can to fulfill the hopes of all those who voted to create the agency when they supported Proposition 71 in 2004.  That’s what this Strategic Plan demonstrates. It’s an ambitious plan, but you never achieve anything worthwhile by playing it safe. Too many lives are at stake for us to do anything less than work as hard as we can, as long as we can, to achieve as much as we can.”

Over the course of the next five years we hope to:

  • Launch 50 new clinical trials covering at least 20 unique diseases or conditions, and including at least 10 rare and 5 pediatric indications
  • Increase the number of projects advancing to the next stage of development by 50%
  • Work with patient advocates, the FDA and researchers to develop a new, more efficient regulatory process for cell therapies
  • Reduce the time it takes a stem cell therapy to move from discovery into a clinical trial by 50%

But wait, there’s more

And that’s just a taste of what we are planning. For the full picture you need to check out the Strategic Plan. But as Randy Mills says, we don’t want you to just read it. This process began with us asking you for your thoughts. Now we want to end it the same way.

“Your input was invaluable in helping us chart an ambitious course and giving us the inspiration to be bold and think outside of the box. Now, as we get ready to put this new vision for the agency into action, we want to share it with the public, with patients and patient advocates, scientists and researchers, and give them a chance to let us know what they think.”

Here’s where you can find the Strategic Plan.

What do you think?

If you have any thoughts or comments send them to me by 5pm, Thursday, December 3rd at kmccormack@cirm.ca.gov

The Strategic Plan is due to go before the CIRM Science Subcommittee on Monday, November 30th and the full Board for its approval on Thursday, December 17th.

 

Improving process drives progress in stem cell research

shutterstock_212888935Process is not a sexy word. No one gets excited thinking about improving a process. Yet behind every great idea, behind every truly effective program is someone who figured out a way to improve the process, to make that idea not just work, but work better.

It’s not glamorous. Sometimes it’s not even pretty. But it is essential.

Yesterday in Oakland our governing Board approved two new concepts to improve our process, to help us fund research in a way that is faster, smarter and ultimately helps us better meet our mission of accelerating the development of stem cell therapies for patients with unmet medical needs.

The new concepts are for Discovery – the earliest stage of research – and the Translational phase, a critical step in moving promising therapies out of the lab and toward clinical trials where they can be tested in people.

In a news release C. Randal Mills, Ph.D., CIRM’s President and CEO, said that these additions built on the work started when the agency launched CIRM 2.0 in January for the clinical phase of research:

“What makes this approach different is that under CIRM 2.0 we are creating a pathway for research, from Discovery to Translational and Clinical, so that if a scientist is successful with their research at one level they are able to move that ahead into the next phase. We are not interested in research just for its own sake. We are interested in research that is going to help us help patients.”

In the Discovery program, for example, we will now be able to offer financial incentives to encourage researchers who successfully complete their work to move it along into the Translational phase – either themselves or by finding a scientific partner willing to take it up and move it forward.

This does a number of things. First it helps create a pipeline for the most promising projects so ideas that in the past might have stopped once the initial study ended now have a chance to move forward. Obviously our hope is that this forward movement will ultimately lead to a clinical trial. That won’t happen with every research program we fund but this approach will certainly increase the possibility that it might.

There’s another advantage too. By scheduling the Discovery and Translational awards more regularly we are creating a grant system that has more predictability, making it easier for researchers to know when they can apply for funding.

We estimate that each year there will be up to 50 Discovery awards worth a total of $53 million; 12 Translation awards worth a total of $40 million; and 12 clinical awards worth around $100 million. That’s a total of more than $190 million every year for research.

This has an important advantage for the stem cell agency too. We have close to $1 billion left in the bank so we want to make sure we spend it as wisely as we can.

As Jonathan Thomas, Ph.D. J.D, the Chair of our Board, said, having this kind of plan helps us better plan our financial future;

“Knowing how often these programs are going to be offered, and how much money is likely to be awarded means the Board has more information to work with in making decisions on where best to allocate our funding.”

The Board also renewed funding for both the Bridges and SPARK (formerly Creativity) programs. These are educational and training programs aimed at developing the next generation of stem cell scientists. The Bridges students are undergraduate or Master’s level students. The SPARK students are all still in high school. Many in both groups come from poor or low-income communities. This program gives them a chance to work in a world-class stem cell research facility and to think about a career in science, something that for many might have been unthinkable without Bridges or SPARK.

Process isn’t pretty. But for the students who can now think about becoming a scientist, for the researchers who can plan new studies, and for the patients who can now envision a potential therapy getting into clinical trials, that process can make all the difference.

Pushing, pulling and dragging stem cell research forward

Government agencies are known for many things, but generally speaking a willingness to do some voluntary, deep self-examination is not one of them. However, for the last few weeks CIRM has been doing a lot of introspection as we develop a new Strategic Plan, a kind of road map for where we are heading.

Patient Advocate meeting in Los Angeles: Photo courtesy Cristy Lytal USC

Patient Advocate meeting in Los Angeles:
Photo courtesy Cristy Lytal USC

But we haven’t been alone. We’ve gone to San Diego, Los Angeles and San Francisco to talk to Patient Advocates in each city, to get their thoughts on what we need to focus on for the future. Why Patient Advocates? Because they are the ones with most skin in the game. They are why we do this work so it’s important they have a say in how we do it.

As Chris Stiehl, a Patient Advocate for type 1 diabetes, said in San Diego: “Let the patient be in the room, let them be part of the conversation about these therapies. They are the ones in need, so let them help make decisions about them right from the start, not at the end.”

A Strategic Plan is, on the surface, a pretty straightforward thing to put together. You look at where you are, identify where you want to go, and figure out the best way to get from here to there. But as with many things, what seems simple on the surface often turns out to be a lot more complicated when looked at in more depth.

The second bit, figuring out where you want to go, is easy. We want to live up to our mission of accelerating the development of stem cells therapies to patients with unmet medical needs. We don’t want to be good at this. We want to be great at this.

Dr. C. Randal Mills talking to Patient Advocates in LA: Photo courtesy Cristy Lytal, USC

Dr. C. Randal Mills talking to Patient Advocates in LA: Photo courtesy Cristy Lytal, USC

The first part, seeing where you are, is a little tougher: it involves what our President and CEO, Dr. Randy Mills, “confronting some brutal facts”, being really honest in assessing where you are because without that honesty you can’t achieve anything.

So where are we as an agency? Well, we have close to one billion dollars left in the bank, we have 12 projects in clinical trials and more on the way, we have helped advance stem cells from a fledgling field to a science on the brink of what we hope will be some remarkable treatments, and we have a remarkable team ready to help drive the field still further.

But how do we do that, how do we identify the third part of the puzzle, getting from where we are to where we want to be? CIRM 2.0 is part of the answer – developing a process to fund research that is easier, faster and more responsive to the needs of the scientists and companies developing new therapies. But that’s just part of the answer.

Some of the Patient Advocates asked if we considered focusing on just a few diseases, such as the ten largest killers of Americans, and devoting our remaining resources to fixing them. And the answer is yes, we looked at every single option. But we quickly decided against that because, as Randy Mills said:

“This is not a popularity contest, you can’t judge need by numbers, deciding the worth of something by how many people have it. We are disease agnostic. What we do is find the best science, and fund it.”

Another necessary element is developing better ways to attract greater investment from big pharmaceutical companies and venture capital to really help move the most promising projects through clinical trials and into patients. That is starting to happen, not as fast as we would like, but as our blog yesterday shows things are moving in this direction.

And the third piece of the pie is getting these treatments through the regulatory process, getting the Food and Drug Administration (FDA) to approve therapies for clinical trials. And this last piece clearly hit a nerve.

Many Patient Advocates expressed frustration at the slow pace of approval for any therapy by the FDA, some saying it felt like they just kept piling up obstacles in the way.

Dr. Mills said the FDA is caught between a rock and a hard place; criticized if it approves too slowly and chastised if it approves too fast, green lighting a therapy that later proves to have problems. But he agreed that changes are needed:

“The regulatory framework works well for things like drugs and small molecules that can be taken in pills but it doesn’t work well for cellular therapies like stem cells. It needs to do better at that.”

One Advocate suggested a Boot Camp for researchers, drilling them in the skills they’ll need to get FDA approval. Others suggested applying political pressure from Patient Advocacy groups to push for change.

As always there are no easy answers, but the meeting certainly raised many great questions. Those are all helping us focus our thinking on what needs to be in the Strategic Plan.

Randy ended the Patient Advocate events by saying the stem cell agency “is in the time business. What we do is time sensitive.” For too many people that time is already running out. We have to do everything we can to change that.

Partnering with Big Pharma to benefit patients

Our mission at CIRM is to accelerate the development of stem cell therapies for patients with unmet medical needs. One way we have been doing that is funding promising research to help it get through what’s called the “Valley of Death.” This is the time between a product or project showing promise and the time it shows that it actually works.

Many times the big pharmaceutical companies or deep pocketed investors, whose support is needed to cover the cost of clinical trials, don’t want to get involved until they see solid proof that this approach works. However, without that support the researchers can’t do the early stage clinical trials to get that proof.

The stem cell agency has been helping get these projects through this Catch 22 of medical research, giving them the support they need to get through the Valley of Death and emerge on the other side where Big Pharma is waiting, ready to take them from there.

We saw more evidence that Big Pharma is increasingly happy doing that this week with the news that the University of California, San Diego, is teaming up with GSK to develop a new approach to treating blood cancers.

Dr. Catriona Jamieson: Photo courtesy Moores Cancer Center, UCSD

Dr. Catriona Jamieson:
Photo courtesy Moores Cancer Center, UCSD

Dr. Catriona Jamieson is leading the UCSD team through her research that aims at killing the cancer stem cells that help tumors survive chemotherapy and other therapies, and then spread throughout the body again. This is work that we have helped fund.

In a story in The San Diego Union Tribune, reporter Brad Fikes says this is a big step forward:

“London-based GSK’s involvement marks a maturation of this aspect of Jamieson’s research from basic science to the early stages of discovering a drug candidate. Accelerating such research is a core purpose of CIRM, founded in 2004 to advance stem cell technology into disease therapies and diagnostics.”

The stem cell agency’s President and CEO, Dr. C. Randal Mills, is also quoted in the piece saying:

“This is great news for Dr. Jamieson and UCSD, but most importantly it is great news for patients. Academic-industry partnerships such as this bring to bear the considerable resources necessary to meaningfully confront healthcare’s biggest challenges. We have been strong supporters of Dr. Jamieson’s work for many years and I think this partnership not only reflects the progress that she has made, but just as importantly it reflects how the field as a whole has progressed.”

As the piece points out, academic researchers are very good at the science but are not always as good at turning the results of the research into a marketable product. That’s where having an industry partner helps. The companies have the experience turning promising therapies into approved treatments.

As Scott Lippman, director of the Moores Cancer Center at UCSD, said of the partnership:

“This is a wonderful example of academia-industry collaboration to accelerate drug development and clinical impact… and opens the door for cancer stem cell targeting from a completely new angle.”

With the cost of carrying out medical research and clinical trials rising it’s hard for scientists with limited funding to go it alone. That’s why these partnerships, with CIRM and industry, are so important. Working together we make it possible to speed up the development and testing of therapies, and get them to patients as quickly as possible.

Share your voice, shape our future

shutterstock_201440705There is power in a single voice. I am always reminded of that whenever I meet a patient advocate and hear them talk about the need for treatments and cures – and not just for their particular disease but for everyone.

The passion and commitment they display in advocating for more research funding reflects the fact that everyday, they live with the consequences of the lack of effective therapies. So as we at CIRM, think about the stem cell agency’s future and are putting together a new Strategic Plan to help shape the direction we take, it only makes sense for us to turn to the patient advocate community for their thoughts and ideas on what that future should look like.

That’s why we are setting up three meetings in the next ten days in San Diego, Los Angeles and San Francisco to give our patient advocates a chance to let us know what they think, in person.

We have already sent our key stakeholders a survey to get their thoughts on the general direction for the Strategic Plan, but there is a big difference between ticking a box and having a conversation. These upcoming meetings are a chance to talk together, to explore ideas and really flesh out the details of what this Strategic Plan could be and should be.

Our President and CEO, Dr. C. Randal Mills wants each of those meetings to be an opportunity to hear, first hand, what people would like to see as we enter our second decade. We have close to one billion dollars left to invest in research so there’s a lot at stake and this is a great chance for patient advocates to help shape our next five years.

Every voice counts, so join us and make sure that yours is heard.

The events are:

San Diego, Monday, July 13th at noon at Sanford Consortium for Regenerative Medicine, 2880 Torrey Pines Scenic Drive, La Jolla, CA 92037

Los Angeles: Tuesday, July 14th at noon at Eli and Edythe Broad CIRM Center for Regenerative Medicine and Stem Cell Research at USC, 1425 San Pablo Street, 1st floor conf. room Los Angeles, CA 90033

San Francisco: Wednesday, July 15th at noon at CIRM, 210 King Street (3rd floor), San Francisco, CA 94107

There will be parking at each event and a light lunch will be served.

We hope to see you at one of them and if you do plan on coming please RSVP to info@cirm.ca.gov

And of course please feel free to share this invitation to anyone you think might be interested in having their voice heard. We all have a stake in this.

Meet the Stem Cell Agency President C. Randal Mills

As you probably have heard by now we have a new President at the stem cell agency. In late April, after a nationwide search, our governing Board appointed C. Randal Mills, PhD, to take over from Alan Trounson who has led the agency for the last six years, and is standing down to spend more time with his family in Australia.

Stem cell agency President C. Randal Mills (left) and Chair of the Board Jonathan Thomas

Stem cell agency President C. Randal Mills (left) and Chair of the Board Jonathan Thomas

Randy comes to us with a track record of success in stem cell research. For the last ten years he has been the President and CEO of Osiris Therapeutics. He helped the company develop Prochymal, used for the treatment of acute graft-vs-host disease in children, a devastating complication of bone marrow transplantation that can be fatal. He is also well known to many of the staff at the stem cell agency, having served as a member of our Grants Working Group review panel for the past five years, helping decide which projects are the most promising and therefore most deserving of funding.

Now we want to introduce him to you.

We are a publicly funded agency so we want to make sure the public gets a chance to know the person who is leading us. That’s why we are holding three events over the next month to give Patient Advocates, researchers, supporters and anyone who is interested a chance to come and hear Randy talk about his vision for the agency and his goals for the future. And of course we want to give you a chance to ask him questions.

Those events are:

    San Francisco: Monday, June 9th from 6 – 7pm at our offices on 210 King Street, San Francisco, CA 94107. We are located between 3rd and 4th streets, there is good muni bus service to the area and we are just one block from the Caltrain Terminal at 700 4th Street.
    Los Angeles: Tuesday, June 10th from 6 – 7pm at the Eli & Edythe Broad CIRM Center for Regenerative Medicine and Stem Cell Research at the Keck School of Medicine of USC. The address there is 1425 San Pablo Street, Los Angeles, CA 90033.
    San Diego: Tuesday, June 24th from 6 – 7pm at the Manchester Grand Hyatt, 1 Market Place, San Diego, CA 92101. This is going to be held at the same time as the BIO 2014 conference so we are meeting in the Grand Hyatt, in the Gaslamp rooms A & B.

At each event we will provide light refreshments.

I do hope you will join us.